Attachment -I Page 1 
SUGGESTION FOR GUIDELINES FOR HUMAN GENE THERAPY. 
Description of genetic condition and requirement for gene therapy. 
General description of approach to gene therapy. 
Evidence of efficacy of particular therapy proposed in animal 
models . 
Details of structure of vector and any other materials to be 
injected or given to humans, that is, if a virus, complete 
description of the structure of the virus and any helper 
viruses present. 
Details of mode of preparation of vector to be injected, that is, 
for a virus, what is the nature of the cells it is grown in 
and any special features of these cells including other DNAs 
present and medium and serum, means of assay for 
contaminating viruses in cells and serum, purification of 
virus, determining structure and purity. 
Evidence no infective particles present in materials to be given to 
humans . 
Mode of delivery and site. 
Evidence of stability of vector in patient, that is, that no 
recombinant or other variant DNAs have appeared and that the 
DNA present in cells iS the same as that Injected. 
Evidence about expression of added DNA, regulation of expression, 
stability of expression, tissue specificity of expression, 
etc . . 
Evidence therapy has no harmful effects. For a virus, that it does 
not cause cancer or harmful mutations or immune response, 
etc.. In particular, evidence no possibility of 
regenerating infectious particles, evidence of- lack of 
oncogeneicity, and evidence of absence of germ-line 
integration . 
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